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Implementing data on targeted therapy from the INFORM registry platform for children with relapsed cancer in Sweden
Sofia Wallin1, Ingrid Øra2, Gabriela Prochazka3
1Division of Pediatric Oncology, Department of Women and Children´s Health, Karolinska Institutet, Stockholm, Sweden.
Background:
Advances in treatment of childhood malignancies have improved overall cure rates to 80%. Nevertheless, cancer is still the most common cause of childhood mortality in Sweden. The prognosis is particularly poor for relapse of high-risk malignancies. In the international INFORM registry, tumor tissue from patients with relapsed, refractory, or progressive pediatric cancer as well as from very-high risk primary tumors is biologically characterized using next-generation sequencing to identify possible therapeutic targets. We analyzed data from Swedish children included in the INFORM registry concerning patient characteristics, survival, sequencing results and whether targeted treatment was administered to the children based on the molecular findings.
Methods:
A registry-based descriptive analysis of 184 patients included in the INFORM registry in Sweden during 2016-2021.
Results:
The most common diagnoses were soft tissue and bone sarcomas followed by high grade gliomas [including diffuse intrinsic pontine glioma (DIPG)]. Complete molecular analysis was successful for 203/212 samples originating from 184 patients. In 88% of the samples, at least one actionable target was identified. Highly prioritized targets, according to a preset scale, were identified in 48 (24%) samples from 40 patients and 24 of these patients received matched targeted treatment but only six children within a clinical trial. No statistically significant benefit in terms of overall survival or progression free survival was observed between children treated with matched targeted treatment compared to all others.
Conclusion:
This international collaborative study demonstrate feasibility regarding sequencing of pediatric high-risk tumors providing molecular data regarding potential actionable targets to clinicians. For a few individuals the INFORM analysis was of utmost importance and should be regarded as a new standard of care with the potential to guide targeted therapy.
Insights
Pediatric cancer patients in the INFORM registry had their tumors sequenced to find treatment targets. While actionable targets were common, matched targeted therapy did not significantly improve survival outcomes in this study.
Area of Science:
- Oncology
- Genomics
- Pediatrics
Background:
- Childhood cancer remains a leading cause of mortality in Sweden, especially for relapsed or high-risk cases.
- The International INFORM registry collects tumor samples from pediatric cancer patients for molecular characterization.
- Next-generation sequencing (NGS) is employed to identify potential therapeutic targets in these tumors.
Purpose of the Study:
- To analyze data from Swedish children in the INFORM registry.
- To investigate patient characteristics, survival rates, and sequencing results.
- To determine if molecular findings guided targeted treatment administration and its impact.
Main Methods:
- A registry-based descriptive analysis was conducted.
- Data from 184 Swedish pediatric cancer patients in the INFORM registry (2016-2021) were analyzed.
- Tumor samples underwent comprehensive molecular analysis using NGS.
Main Results:
- Soft tissue/bone sarcomas and high-grade gliomas were the most frequent diagnoses.
- Actionable molecular targets were identified in 88% of analyzed samples.
- 24 patients received matched targeted treatment, but no significant improvement in overall or progression-free survival was observed compared to other treatments.
Conclusions:
- The INFORM study demonstrates the feasibility of sequencing pediatric high-risk tumors.
- Molecular data from sequencing can provide potential actionable targets for clinicians.
- While not universally beneficial in this cohort, INFORM analysis holds potential as a standard of care to guide targeted therapies for select individuals.
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